PB0200 - Clinical outcomes from treatment with extended half-life and standard half-life products in a hemophilia patients’ cohort in Colombia

Background: Hemophilia is a coagulation factor VIII or IX deficiency causing bleeding that can be life‐threatening or lead to debilitating long‐term complications (1,2). There are several standard half-life (SHL) and extended half-life (EHL) products with comparable efficacy and safety.

Aims: To characterize the clinical outcomes in a patients’ cohort with hemophilia A or B treated with SHL or EHL factors in a hemophilia treatment center (HTC) in Colombia.

Methods: Retrospective cohort study of patients with hemophilia A or B from February 2014 until August 2023. Annualized bleeding rate (ABR), type of treatment (episodic vs prophylactic) and bleeding localization were estimated with the total number of bleedings according to time at risk (since program entry to the first registered bleeding) in the cohort and according to the EHL or SHL factor received were evaluated from clinical records. Quality of life was assessed through a questionnaire.

Results: Out of 927 patients, 89.1% (n=826) were men, aged 29 years (IR 17 to 43), with a 19.9 years-median time since diagnosis (IR 11.3 to 33.5). 79.7% had hemophilia A, being 34.8%(n=323) mild, 23.8%(n=221) moderate, 41,1% (n=381) severe and 0.22%(n=2) without classification. The median factor level was 2.5% (IR 0.4-10). 52.2%(n=484) were on episodic treatment, 25.0%(n=232) on prophylaxis, and 22.7%(n=211) without or unknown treatment. From the total cohort 17.2%(159) had any bleeding, from whom 67.9%(108/159) accounted for hemarthrosis, muscle and soft tissue hemorrhages in 42.8%(68/159) and 3.1(5/159) iliopsoas. In hemarthrosis 24.2%(22/91) were spontaneous and 89%(81/91) traumatic. The bleeding rate was 13.7 per 100 person-years at risk (95%CI 11.6-16.2), for EHL 17.2 (95%CI 10.4-28.5) and SHL 13.4 (95%CI 11.2-16.0) (Table 1). In the prophylaxis group, the bleeding rate was 10.7 while in the demand was 14.5x100 person-years.

Conclusion(s): Severe patients with EHL surprisingly had a higher bleeding rate than those with SHL, represented mainly by hemarthrosis.